Picture CRISPR-Cas9, a gene editing technology, as a GPS-guided scalpel: gRNA directs the Cas9 enzyme, a protein that cuts ...
Genetic disorders occur due to alterations in the primary genetic material—deoxyribonucleic acid (DNA)—of an organism.
Despite emerging clinical successes, current genome editors suffer from off-target effects and can trigger unwanted responses from the immune system, limiting their broader therapeutic applications.
A major medical milestone took place in May 2025, when doctors at the Children’s Hospital of Philadelphia used CRISPR-based gene editing to treat a child with a rare genetic disorder. Unlike earlier ...
Researchers at Utah State University revealed new details about CRISPR immune system defenses, such as Cas12a3 systems, that ...
In a 15-patient, Phase 1, first-in-human trial, a one-time CRISPR-Cas9 gene-editing therapy safely reduced LDL cholesterol and triglycerides in people with difficult-to-treat lipid disorders, ...
Stanford Medicine researchers have developed an artificial intelligence tool to help scientists better plan gene-editing experiments. The technology, CRISPR-GPT, acts as a gene-editing "copilot" ...